Publication date: Mar 27, 2025
Patients experience debilitating symptoms, including severe orthostatic hypotension, urinary dysfunction, ataxic gait, dysmetria, scanning speech, and cognitive decline, significantly impacting their quality of life. Current therapies focus on symptomatic management, such as dopamine therapy and medications for autonomic dysfunction, but often yield disappointing results. To date, no approved agents are available for MSA. About YA-101YA-101 is an AI-pioneered NCE with a dual modulation mechanism specifically designed to treat neurodegenerative diseases. This Phase 2 trial is a randomized, double-blind, placebo-controlled investigation for MSA patients with walking ability, including those who need walking assistance. Measurement of clinical symptoms, biomarkers, and imaging will allow a comprehensive assessment. Further details are available at https://clinicaltrials. gov/study/NCT06848231. On July 1, 2022, it received Orphan Drug Designation (ODD) for MSA from the United States Food and Drug Administration (FDA).
| Concepts | Keywords |
|---|---|
| March | Atrophy |
| Parkinsonism | Clinical |
| Pharmacokinetics | Disorder |
| Taipei | Dysfunction |
| Motor | |
| Msa | |
| Multiple | |
| Neurodegenerative | |
| Patient | |
| Phase | |
| Rare | |
| System | |
| Trial | |
| United | |
| Yoda |
Semantics
| Type | Source | Name |
|---|---|---|
| disease | MESH | ataxia |
| drug | DRUGBANK | Dopamine |
| disease | MESH | cognitive decline |
| disease | MESH | scanning speech |
| disease | MESH | gait |
| disease | MESH | orthostatic hypotension |
| disease | MESH | neuroinflammation |
| pathway | REACTOME | Neurodegenerative Diseases |
| disease | MESH | cerebellar ataxia |
| disease | MESH | parkinsonism |
| disease | MESH | causes |
| disease | MESH | neurodegenerative disorder |
| drug | DRUGBANK | Nonoxynol-9 |
| disease | MESH | Multiple System Atrophy |